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Single-dose, subcutaneous recombinant phenylalanine ammonia lyase conjugated with polyethylene glycol in adult patients with phenylketonuria: an open-label, multicentre, phase 1 dose-escalation trial

The Lancet (British edition), 2014-07, Vol.384 (9937), p.37-44

2014

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Safety, tolerability and pharmacodynamics of apical sodium-dependent bile acid transporter inhibition with volixibat in healthy adults and patients with type 2 diabetes mellitus: a randomised placebo-controlled trial

BMC gastroenterology, 2018-01, Vol.18 (1), p.3-17, Article 3

2018

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Maralixibat for the treatment of PFIC: Long‐term, IBAT inhibition in an open‐label, Phase 2 study

Hepatology communications, 2022-09, Vol.6 (9), p.2379-2390

2022

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Impact of Asthma Exacerbations and Asthma Triggers on Asthma-related Quality of Life in Patients with Severe or Difficult-to-Treat Asthma

The journal of allergy and clinical immunology in practice (Cambridge, MA), 2014-09, Vol.2 (5), p.544-552.e2

2014

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Treatment of VLCAD-Deficient Patient Fibroblasts with Peroxisome Proliferator-Activated Receptor δ Agonist Improves Cellular Bioenergetics

Cells (Basel, Switzerland), 2022-08, Vol.11 (17), p.2635

2022

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Clinical Burden and Predictors of Asthma Exacerbations in Patients on Guideline-based Steps 4-6 Asthma Therapy in the TENOR Cohort

The journal of allergy and clinical immunology in practice (Cambridge, MA), 2014-03, Vol.2 (2), p.193-200.e3

2014

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Assessment of asthma control and asthma exacerbations in the epidemiology and natural history of asthma: outcomes and treatment regimens (TENOR) observational cohort

Current respiratory care reports, 2012-12, Vol.1 (4), p.259-269

2012

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Efficacy and safety of maralixibat treatment in patients with Alagille syndrome and cholestatic pruritus (ICONIC): a randomised phase 2 study

The Lancet (British edition), 2021-10, Vol.398 (10311), p.1581-1592

2021

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A Randomized, Controlled, Phase 2 Study of Maralixibat in the Treatment of Itching Associated With Primary Biliary Cholangitis

Hepatology communications, 2019-03, Vol.3 (3), p.365-381

2019

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PS-193-Phase 2 open-label study with a placebo-controlled drug withdrawal period of the apical sodium-dependent bile acid transporter inhibitor maralixibat in children with Alagille Syndrome: 48-week interim efficacy analysis

Journal of hepatology, 2019-04, Vol.70 (1), p.e119-e120

2019

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A Recombinant Antibody to Siglec-8 Shows Selective ADCC Activity Against Mast Cells from Systemic Mastocytosis Patients

Blood, 2015-12, Vol.126 (23), p.4092-4092

2015

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Two-dose intrapartum/newborn nevirapine and standard antiretroviral therapy to reduce perinatal HIV transmission: a randomized trial

JAMA : the journal of the American Medical Association, 2002-07, Vol.288 (2), p.189

2002

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Development of a Novel Tool to Assess the Impact of Itching in Pediatric Cholestasis

The patient : patient-centered outcomes research, 2018-02, Vol.11 (1), p.69-82

2018

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Safety, tolerability, and efficacy of maralixibat in adults with primary sclerosing cholangitis: Open-label pilot study

Hepatology communications, 2023-06, Vol.7 (6)

2023

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Rhinology Future Debates, an EUFOREA Report

Rhinology, 2017-01, Vol.55 (4), p.298-304

Fokkens, Wytske

2017

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Phase I Safety and Pharmacokinetics Study of Micronized Atovaquone in Human Immunodeficiency Virus-Infected Infants and Children

Antimicrobial Agents and Chemotherapy, 1998-06, Vol.42 (6), p.1315-1318

1998

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Maternal toxicity and pregnancy complications in human immunodeficiency virus–infected women receiving antiretroviral therapy: PACTG 316

American journal of obstetrics and gynecology, 2004-02, Vol.190 (2), p.506-516

2004

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When the time comes to talk about HIV: factors associated with diagnostic disclosure and emotional distress in HIV-infected children

Journal of acquired immune deficiency syndromes (1999), 2002-11, Vol.31 (3), p.309

2002

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Efficacy and safety of heparinase I versus protamine in patients undergoing coronary artery bypass grafting with and without cardiopulmonary bypass

Anesthesiology (Philadelphia), 2005-08, Vol.103 (2), p.229-240

2005

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Nevirapine : Pharmacokinetic considerations in children and pregnant women

Clinical pharmacokinetics, 2000-10, Vol.39 (4), p.281-293

2000

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Predose infant nevirapine concentration with the two-dose intrapartum neonatal nevirapine regimen: association with timing of maternal intrapartum nevirapine dose

Journal of acquired immune deficiency syndromes (1999), 2003-06, Vol.33 (2), p.153

2003

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Characteristics and management of HIV-1-infected pregnant women enrolled in a randomised trial: differences between Europe and the USA

BMC infectious diseases, 2007-06, Vol.7 (1), p.60-60, Article 60

2007

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Sleep disturbances in children with human immunodeficiency virus infection

Pediatrics (Evanston), 1999-11, Vol.104 (5), p.e62

1999

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Diagnostic Disclosure to HIV-Infected Children: How Parents Decide when and what to Tell

Clinical child psychology and psychiatry, 2002-01, Vol.7 (1), p.85-99

2002

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Chaperone-Like Therapy with Tetrahydrobiopterin in Clinical Trials for Phenylketonuria: Is Genotype a Predictor of Response?

JIMD Reports - Case and Research Reports, 2012/2, 2012, Vol.5, p.59-70

2012

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Single-dose, subcutaneous recombinant phenylalanine ammonia lyase conjugated with polyethylene glycol in adult patients with phenylketonuria: an open-label, multicentre, phase 1 dose-escalation trial

Safety, tolerability and pharmacodynamics of apical sodium-dependent bile acid transporter inhibition with volixibat in healthy adults and patients with type 2 diabetes mellitus: a randomised placebo-controlled trial

Maralixibat for the treatment of PFIC: Long‐term, IBAT inhibition in an open‐label, Phase 2 study

Impact of Asthma Exacerbations and Asthma Triggers on Asthma-related Quality of Life in Patients with Severe or Difficult-to-Treat Asthma

Treatment of VLCAD-Deficient Patient Fibroblasts with Peroxisome Proliferator-Activated Receptor δ Agonist Improves Cellular Bioenergetics

Clinical Burden and Predictors of Asthma Exacerbations in Patients on Guideline-based Steps 4-6 Asthma Therapy in the TENOR Cohort

Assessment of asthma control and asthma exacerbations in the epidemiology and natural history of asthma: outcomes and treatment regimens (TENOR) observational cohort

Efficacy and safety of maralixibat treatment in patients with Alagille syndrome and cholestatic pruritus (ICONIC): a randomised phase 2 study

A Randomized, Controlled, Phase 2 Study of Maralixibat in the Treatment of Itching Associated With Primary Biliary Cholangitis

PS-193-Phase 2 open-label study with a placebo-controlled drug withdrawal period of the apical sodium-dependent bile acid transporter inhibitor maralixibat in children with Alagille Syndrome: 48-week interim efficacy analysis

A Recombinant Antibody to Siglec-8 Shows Selective ADCC Activity Against Mast Cells from Systemic Mastocytosis Patients

Two-dose intrapartum/newborn nevirapine and standard antiretroviral therapy to reduce perinatal HIV transmission: a randomized trial

Development of a Novel Tool to Assess the Impact of Itching in Pediatric Cholestasis

Safety, tolerability, and efficacy of maralixibat in adults with primary sclerosing cholangitis: Open-label pilot study

Rhinology Future Debates, an EUFOREA Report

Phase I Safety and Pharmacokinetics Study of Micronized Atovaquone in Human Immunodeficiency Virus-Infected Infants and Children

Maternal toxicity and pregnancy complications in human immunodeficiency virus–infected women receiving antiretroviral therapy: PACTG 316

When the time comes to talk about HIV: factors associated with diagnostic disclosure and emotional distress in HIV-infected children

Efficacy and safety of heparinase I versus protamine in patients undergoing coronary artery bypass grafting with and without cardiopulmonary bypass

Nevirapine : Pharmacokinetic considerations in children and pregnant women

Predose infant nevirapine concentration with the two-dose intrapartum neonatal nevirapine regimen: association with timing of maternal intrapartum nevirapine dose

Characteristics and management of HIV-1-infected pregnant women enrolled in a randomised trial: differences between Europe and the USA

Sleep disturbances in children with human immunodeficiency virus infection

Diagnostic Disclosure to HIV-Infected Children: How Parents Decide when and what to Tell

Chaperone-Like Therapy with Tetrahydrobiopterin in Clinical Trials for Phenylketonuria: Is Genotype a Predictor of Response?

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